{"id":"doi:10.3390/jcm11226773","kind":"review","title":"Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review","authors":["Fu Q","Hu L","Shen T","Yang R","Jiang L"],"author_count":5,"year":2022,"published":"2022-11-16","venue":"Journal of clinical medicine","cited_by":29,"doi":"10.3390/jcm11226773","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.3390/jcm11226773","archive_url":"https://space-kitty.com/papers/doi:10.3390/jcm11226773","updated":null,"pmid":"36431249","pmcid":"PMC9699383","openalex_id":null,"pdf_url":"https://europepmc.org/articles/PMC9699383/pdf","abstract":"(1) Background: Existing lipid-lowering therapies have difficulty in achieving lipid target levels in patients with familial hypercholesterolemia (FH), especially in the treatment of patients with homozygous familial hypercholesterolemia. (2) Method: All of the literature data containing \"Familial hypercholesterolemia\" and \"Gene Therapy\" in PubMed and Clinical Trials from 2018 to 2022 were selected. (3) Results: The rapid development of gene therapy technology in recent years is expected to change the treatment status of FH patients. As emerging gene therapy vectors, the optimized adeno-associated viruses, exosomes, and lipid nanoparticles have demonstrated an improved safety and higher transfection efficiency. Various RNA-targeted therapies are in phase 1-3 clinical trials, such as small interfering RNA-based drugs inclisiran, ARO-ANG3, ARO-APOC3, olpasiran, SLN360, and antisense oligonucleotide-based drugs AZD8233, vupanorsen, volanesorsen, IONIS-APO(a)Rx, etc., all of which have demonstrated excellent lipid-lowering effects. With gene editing technologies, such as CRISPR-Cas 9 and meganuclease, completing animal experiments in mice or cynomolgus monkeys and demonstrating lasting lipid-lowering effects, patients with FH are expected to reach a permanent cure in the future. (4) Conclusion: Gene therapy is being widely used for the lipid-lowering treatment of FH patients and has shown excellent therapeutic promise, but the current delivery efficiency, economic burden, immunogenicity and the precision of gene therapy can be further optimized.","abstract_license":"CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)","license":"CC-BY","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":[],"details":null,"provenance":"europepmc","attribution":"Abstract of \"Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review\" by Fu Q, Hu L, Shen T et al., https://doi.org/10.3390/jcm11226773, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"cardiovascular-innovation","name":"Cardiovascular Therapeutics","domain_slug":"medicine"}],"cited_in_dossiers":[]}